What is the greatest issue facing Parkinson’s patients today, and what can be done to accelerate the research and development of new therapies to help them? 

People who live with Parkinson’s (PwP) today are faced with the inevitability of the neurodegenerative nature of their condition – a slow loss of control over life as time progresses. All ambitions and expectations for the future become compromised, and the resulting sense of loss becomes increasingly burdensome. The principal medicine for this is encapsulated in one word – hope. Unfortunately, hope is not a word often used or associated with science – hope is uncertain and science relies on evidence of certainty.

To translate hope into scientific terms, there must be something tangible and real. From the moment of diagnosis, the sand in the hourglass starts to run and, as time passes, the urgency required to maintain hope intensifies.

In plain English, for most people with Parkinson’s the only way the hopes for new treatments can be converted into belief of new treatments, and then onto realisation, is through the conduct of clinical trials. It stands to reason, therefore, that PwP’s hopes are almost totally vested in the conduct, management and success of clinical trials.

Barriers to advancing clinical trials

The inordinate amount of time, money and regulatory hurdles in bringing any new drug to market in any condition are well documented, but there are some specific problems in Parkinson’s. One of the issues facing industry when assessing whether to develop a new Parkinson’s treatment is measurement. Parkinson’s is different in every PwP and there can be huge fluctuations in symptoms within very short time spans. The lack of an objective, continuous and accurate method of assessing PwP means that the results and statistical analysis of clinical trials become difficult to interpret and, quite often, are inconclusive. This, coupled with costs and the length of time needed to show clinically significant changes in the trial participants’ symptoms, too often renders Parkinson’s too risky a business case for pharmaceutical companies.

Nevertheless, there are now several new scientific avenues which are being explored as targets for the development of therapies with good prospects of delaying, stopping or even reversing the condition. But with the costs of conducting robust and meaningful studies of any one of these being so prohibitively high, there is still huge reluctance for industry to enter the Parkinson’s arena for reasons of commercial viability alone. What is needed, therefore, is a means of de-risking investment by means of a scientific screening process.

A Way Forward

In 2012 The Cure Parkinson’s Trust (CPT) instigated its Linked Clinical Trials initiative (LCT). The objective was to apply new scientific and clinical knowledge gained about the condition and to find ways of addressing the barriers which stand in the way of translating new science into new treatments.

Linked Clinical Trials

In 2012 and 2013 CPT compiled comprehensive dossiers of over a hundred prospective therapeutic targets for Parkinson’s, all of which have the necessary safety profile to be administered to people living with the condition now. These dossiers were scrutinised and prioritised by a specially selected group of nine of the world’s most renowned Parkinson’s specialists (‘The Committee’) at meetings held in Grand Rapids, Michigan and Cumberland Lodge, Windsor respectively. A shortlist was drawn up of the drugs with the most likely prospects of slowing, stopping and/or reversing the progression of the disease.

The idea behind this initiative is to accelerate recent breakthroughs in Parkinson’s, and to reduce the costs and time incurred in ‘standard’ trials. This is achieved by focusing on specific data from the trial which is sufficiently robust to determine whether further investigation and financial investment are merited in subsequent large-scale studies. Also, the structure of LCT is such that it allows direct comparison between prospective therapies by harmonising trial designs and outcomes.

In recognition of the importance of this project, CPT intends to pool resources with other funding bodies and expertise to accelerate the progress of this exciting and cutting-edge clinical initiative with a sense of real urgency.

- See more at: http://www.phrma.org/conversations/greatest-issue-facing-parkinson%E2%80%99s-patients-today# and join the conversation!

PhRMA’s mission to effectively advocate for public policy that encourages innovative drug development, drug discovery and drug research in the biopharmaceutical industry would not be possible without participation and interest from people like you. See
 for further information.